A cutting-edge therapy using base-edited immune cells is offering a major breakthrough for patients with one of the toughest ...

The Food and Drug Administration this week granted approval to a new gene therapy for a rare immune disorder, the maker of which is notably not a drug ...

Sarepta Therapeutics (NASDAQ:SRPT) leads in genetic therapies, contributing to ongoing discussions surrounding the nasdaq ...

Backed by Italy-based Fondazione Telethon ETS, Waskyra, for Wiskott-Aldrich syndrome, is the first gene therapy from a ...

EpilepsyGTx, a biotechnology company focused on research and development of cutting-edge gene therapies to treat refractory epilepsy, today announced it has raised $33 million in a Series A financing ...

Gungnir is launching an animation studio that will feed an in-house YouTube channel that planned as "the Cartoon Network of ...

Rocket Pharmaceuticals (NASDAQ:RCKT) advances gene-therapy innovation and appears in broader discussions involving the nasdaq ...

In this video from the American Academy of Ophthalmology meeting, Jennifer I. Lim, MD, MBA, discusses three gene therapies currently being studied for the treatment of age-related macular degeneration ...

A cutting-edge base-edited CAR T-cell therapy is opening a revolutionary new frontier for patients battling aggressive T-cell ...

A single-dose gene replacement therapy is found to improve movement ability in children over 2 years of age and teenagers with spinal muscular atrophy, according to research published in Nature ...

On a video call in early September, Sarah Tabrizi first saw the data that she and other researchers studying Huntington’s ...

NEW ORLEANS — New data from a first-in-human trial of a CRISPR-based gene therapy for cholesterol disorders are giving lipids experts reason for optimism about the approach for treating cardiovascular ...